IND Application Submission To FDA For Phase 1 Trial Of Genetically Modified Autologous Cell Therapy For HIV Announced by American Gene Technologies

IND Application Submission To FDA For Phase 1 Trial Of Genetically Modified Autologous Cell Therapy For HIV Announced by American Gene Technologies

ROCKVILLE, Md.Oct. 18, 2019 /PRNewswire/ -- American Gene Technologies (AGT) announced today the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for AGT's lead HIV program, AGT103-T, which is potentially a single-dose, lentiviral vector-based gene therapy developed for the purpose of eliminating HIV from people infected with the disease.

"Our aim is to treat HIV disease with an innovative cell and gene therapy that reconstitutes immunity to HIV and will control virus growth in the absence of antiretroviral drugs" said Chief Science Officer C. David Pauza, PhD. "Development of this complex product (AGT103-T) required our deep knowledge of both HIV disease and lentivirus vector technology; it is the first cell and gene immunotherapy addressing the most critical feature of HIV infection, which is the chronic absence of virus-specific CD4 T cells."

Dr. David Pauza Video

"We are excited to have reached this milestone of submitting our first IND application to the FDA for an HIV gene/cell therapy. This event brings us closer to reaching our mission to transform lives with genetic medicines. Based on our successful commercial-scale product manufacturing runs and features of the product observed in our laboratories, this therapy has a high potential to be effective. I feel confident that AGT103-T will make an important difference in the lives of HIV infected persons," said Jeff Galvin, Founder and Chief Executive Officer of AGT. "HIV is the first drug candidate to result from AGT's proprietary platform and model for creating gene and cell therapeutics more efficiently, predictably and reliably for clinical development. Our platform is also supporting robust efforts to cure the inherited disease phenylketonuria (PKU) and to introduce new therapies for cancer based on our proprietary methods for modifying tumor cells to activate the natural killing mechanisms of gamma delta T cells. Additionally, product development efforts behind this IND submission are supporting investigations into other chronic viral diseases that may be targeted in future human clinical trials. Success in HIV would allow AGT to accelerate these projects as well as quickly broaden our pipeline to dozens of infectious diseases, monogenic disorders, and cancers."

Upon acceptance by the FDA, this IND allows AGT to initiate a Phase 1 clinical trial that will investigate the safety of AGT103-T in humans, measure key biomarkers, and explore surrogate markers of efficacy. AGT expects to begin recruiting patients for the Phase 1 study in January.

About HIV

Today, approximately 37.9 million people worldwide and 1.1 million people in the United States are living with HIV/AIDS. The U.S. Government has estimated that 38,700 Americans were newly infected with HIV in 2016 and 1.7 million individuals globally were newly infected with HIV in 2018 (

Since the late 1980s, antiretroviral drugs have restored quality of life to persons living with HIV and, in some cases, have even been used to prevent new infections. However, no approved treatments can cure HIV. This is an unmet medical need that AGT seeks to address.

About AGT 103-T

AGT103-T is a genetically-modified cell product made from a person's own cells. AGT's approach is unique in that it focuses on repairing the key immune system damage caused by HIV. When HIV infection causes this specific damage, killing of T helper cells required for immunity to HIV, the infected person becomes unable to eliminate the virus and thus, becomes chronically infected. AGT's approach is designed to repair the T helper cell defect and provide durable virus control that is not compromised by HIV strains that vary in sequence or use alternate ways to enter and infect T cells. AGT's AGT103-T HIV therapeutic drug should work to remove infected cells from the body and decrease or eliminate the need for lifelong antiretroviral treatment.

"Previous cell and gene therapies for HIV provided very low doses of critical virus-specific CD4 T cells that are needed to repair the immune defect caused by HIV. AGT103-T becomes highly enriched in these specific cells during our proprietary 12-day manufacturing process. By providing high doses of virus-specific helper T cells, which are protected from HIV damage by a safe genetic modification, AGT's goal is to rebuild the capacity for normal, unhindered immune responses against HIV that may control the infection and protect against future virus exposures. We believe this product will be suitable for persons in different disease stages and with multiple types of HIV infection" explains Chief Science Officer C. David Pauza, PhD.

AGT has entered into a Research Collaboration Agreement with the National Institute of Allergy and Infectious Diseases (NIAID) and was able to demonstrate AGT103-T's mechanism of action. Read about AGT's NIAID Research Collaboration Agreement here.

About American Gene Technologies (AGT)

American Gene Technologies (AGT) is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. The Company's mission is to transform people's lives through genetic medicines that rid the body of disease. The Company expects to take its patented lead candidate for an HIV cure into the clinic in 2019. AGT has received seven patents for its unique immuno-oncology approach to stimulate gamma-delta (γδ) T cells to destroy a variety of solid tumors. The Company has developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. AGT's treatment for PKU has been granted Orphan Drug Designation by the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2020.

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